Martinelli, Giovanni, Cilloni, Daniela, Ottaviani, Emanuela, Malagola, Michele, Messa, Francesca, Rondoni, Michela, Bosi, Costanza, Gottardi, Enrico, Rosti, Gianantonio, Ricci, Paolo, Gaitani, Stavroula, Pane, Fabrizio, Testoni, Nicoletta, Mecucci, Cristina, Soverini, Simona, Piccaluga, Pier Paolo, Amabile, Marilina, Tiribelli, Mario, Zaccaria, Alfonso, Grafone, Tiziana, De Vivo, Antonello, Pileri, Stefano, Fattori, Pierpaolo, Bocchia, Monica, Serra, Anna, Maurillo, Luca, Fanin, Renato, Cross, Nicholas C.P., Merante, Serena, Cazzola, Mario, Alimena, Giuliana, Frassoni, Francesco, Galieni, Piero, Russo, Domenico, Gobbi, Marco, Gugliotta, Luigi, Lauria, Francesco, Mazza, Patrizio, Ferrara, Felicetto, Gherlinzoni, Filippo, Leoni, Pietro, Musto, Pellegrino, Baccarani, Michele and Saglio, Giuseppe
Idiopathic hypereosinophilic syndrome (HES) with FIP1L1-PDGFRA rearrangement can be effectively treated with Imatinib
Blood, 104, (11), .
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We studied 141 patients with HES, of which 55 primary HES (39%) defined as a peripheral blood eosinophilia greater than 1,500 cells/µL for longer than 6 months, absence of other apparent aetiologies for eosinophilia and symptoms of organ involvement. All patients were studied by molecular analysis for PDGFRB-TEL, FGFR1-BCR and BCR-ABL transcripts, frequently associated with HES/CMML/MDS syndrome: all these transcripts were absent in our series. We also sought for the recently reported involvement of PDGFR, cryptically translocated with FIP1L1 in some HES pts responsive to Imatinib therapy: 13 pts (23%) were positive for the FIP1L1-PDGFRA rearrangement and all of them showed previously unreported, abnormal-sized fusion transcripts. Curiously, all FIP1L1-PDGFRA positive pts were male. We enrolled in a national clinical trial 31 (55%) primary HES pts, including all 13 (23%) FIP1L1-PDGFRA positive, with imatinib mesylate (100 to 400 mg/day). Median follow up of treatment was 4,5 moths (range 2–28). Rapid and complete haematological responses to imatinib therapy were recorded only in all FIP1L1-PDGFRA positive pts (100%) after one months of therapy and partial response in only one cases with HES without FIP1L1-PDGFRA fusion transcript. Complete molecular response without evidence of FIP1L1-PDGFRA transcript by qualitative RT-PCR was also recorded in all responding pts after median 2 months of therapy. We conclude that FIP1L1-PDGFRA rearrangement may be useful molecular marker of myeloproliferative HES, a predictor of imatinib-responsiveness and as a means to follow therapy in this subgroup of pts.
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